News

The experimental therapy IONIS-PKK-LRx can significantly lower the number of monthly swelling attacks in patients with hereditary angioedema (HAE) type 1 and 2, according to top-line data from an ongoing Phase 2 trial. Newly announced data show the study (NCT04030598), sponsored by therapy developer Ionis Pharmaceuticals,…

As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking.  But exactly how expensive — in terms of direct and indirect costs across rare disease populations — might still come as a surprise: almost…

Haegarda, CSL Behring’s preventive treatment for hereditary angioedema (HAE) attacks, will be available to eligible HAE patients across Canada starting on April 6. The availability announcement was issued by Canadian Blood Services (CBS), the country’s blood authority, which is responsible for managing the blood supply in all…

Off-label C1 inhibitor concentrate successfully treated people with severe acute angioedema triggered by the use of so-called ACE inhibitor medications, a small study has found. The study, “Effectiveness of C1-INH therapy in angiotensin converting…

Intellia Therapeutics is advancing clinical manufacturing activities to support its plans to file regulatory applications for first-in-human clinical trials of NTLA-2002, its experimental gene-editing therapy for hereditary angioedema (HAE). The submissions are anticipated in the second half of the year, and the first trial is expected to evaluate…

Orladeyo (berotralstat) — an oral therapy developed by BioCryst Pharmaceuticals to prevent swelling attacks in people with hereditary angioedema (HAE) — is now available to patients, 12 and older, in France, following a decision by a government agency. The French National Agency for Medicines and Health Products Safety granted…

Takeda has submitted an application in Japan seeking regulatory approval of Takhzyro (lanadelumab) for the treatment of hereditary angioedema (HAE). The company’s new drug application to the Japanese Ministry of Health, Labour and Welfare (MHLW) specifically asks Takhzyro be approved for the prophylactic, or preventive, treatment of…

The first patient has been dosed in RAPIDe-1, a Phase 2 trial of PHVS416, an oral on-demand treatment being developed by Pharvaris for swelling attacks in people with hereditary angioedema (HAE). RAPIDe-1 (NCT04618211) will evaluate the investigational therapy’s safety, efficacy, and pharmacokinetics, or how the compound moves…

Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, blue, and purple lights side-by-side? These are the colors most often used to represent Rare Disease Day. This…

BioCryst Pharmaceuticals is seeking the approval of Orladeyo (berotralstat) in the United Kingdom as a preventive treatment for sudden and recurrent swelling attacks in people with hereditary angioedema (HAE), ages 12 and older. If granted, Orladeyo would become the first oral therapy — found in trials to be…